New hope for rare disease: first human test of FLT201 begins
NCT ID NCT05324943
First seen Nov 05, 2025 · Last updated May 12, 2026 · Updated 26 times
Summary
This early-stage study tested a new drug, FLT201, in 6 adults with Gaucher disease type 1. The main goal was to check safety and how well the body tolerates the drug. Researchers also looked at whether FLT201 can boost the missing enzyme and reduce harmful buildup in cells.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GAUCHER DISEASE, TYPE 1 are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Hospital Quironsalud Zaragoza
Zaragoza, Spain
-
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
-
Kaiser Permanente
Los Angeles, California, 90027, United States
-
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
-
Rabin Medical Center - PPDS
Petah Tikva, Israel
-
Royal Free Hospital
London, United Kingdom
-
Salford Royal Hospital
Salford, United Kingdom
-
Shaare Zedek Medical Center
Jerusalem, Israel
-
SphinCS
Höchheim, Germany
-
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
Conditions
Explore the condition pages connected to this study.